Sankt Augustin

Researchers are evaluating the long-term safety of subcutaneous guselkumab injections in children with moderately to severely active ulcerative colitis, Crohns disease, or juvenile psoriatic arthritis. This Phase 3 study focuses on pediatric participants who have previously been treated with guselkumab and will continue therapy in this extension study to monitor safety over an extended period. Participants who completed dosing in one of three primary pediatric guselkumab studies and are deemed by their investigator to benefit from continued treatment will join this long-term extension. Guselkumab is administered as a subcutaneous injection either every 8 weeks or every 4 weeks, depending on prior study assignment and clinical status. Some participants may switch dosing frequency once during the extension before unblinding, after which dosing aligns with their original regimen. Dose adjustments are restricted based on the primary study they came from. During the study, participants will receive guselkumab injections regularly and be monitored for treatment-emergent adverse events for up to nearly seven years. Researchers will assess safety outcomes through ongoing clinical evaluations over this time. Parents or legal representatives provide consent for children to participate, and children capable of understanding the study will give assent. The total participation duration may extend up to six years and nine months, allowing long-term safety data collection.

Researchers are conducting a phase II trial to evaluate the clinical benefit, safety, and tolerability of MAS825 arumakimig in both children and adults diagnosed with Stills disease who have not responded adequately to IL-1 andor IL-6 or other available treatments. The study also includes participants with associated lung disease or macrophage activation syndrome. This trial is open-label and single-arm, aiming to assess MAS825 in this patient group. Participants will receive the experimental drug MAS825 during two treatment periods following an initial screening phase. The total study duration for each participant is approximately 16 months. The trial is designed with three periods Screening, Treatment Period 1, and Treatment Period 2. MAS825 administration is the focus throughout the treatment periods, with no placebo or comparator groups. During the study, participants will undergo assessments including clinical response evaluations based on specific criteria by Day 85, biomarker level measurements, physician assessments, and quality of life evaluations. Researchers will monitor changes in glucocorticoid dosage and disease activity over time. Safety and tolerability are closely followed, and inactive disease status while on low-dose corticosteroids will be assessed by 15 months. The study is sponsored by Novartis Pharmaceuticals and includes both pediatric and adult participants aged 1 to 100 years.

Researchers are studying the use of sarilumab, a drug given by injection, in children and adolescents aged 1 to 17 years who have systemic juvenile idiopathic arthritis sJIA. The study aims to understand how the drug behaves in the body, its effects, and its long-term safety for treating this condition. This trial is a phase 2, open-label study sponsored by Sanofi, designed to find the best dose and treatment schedule for young patients with sJIA. Participants will receive sarilumab injections under the skin at doses that increase during the study based on body weight. The treatment includes a 12-week core phase where patients receive the drug, followed by a 144-week extension phase for continued treatment. After completing treatment, a 6-week follow-up period will monitor patients. The study includes careful dose adjustments and long-term observation to assess the drugs impact. Throughout the study, participants will undergo various assessments including blood tests to measure drug levels, evaluations of disease activity using scales like the Investigator Global Assessment and ParentPatient Global Assessment, and tracking of symptoms and medication use. Safety will be monitored continuously by recording any side effects or local reactions to injections. The total participation time is about 166 weeks, during which the researchers will collect data on how well sarilumab works and how safe it is for children and adolescents with sJIA.

Researchers are studying baricitinib to see if it can help preserve beta-cell function in children and adults aged 1 to 35 years who have been newly diagnosed with type 1 diabetes. This Phase 3 study aims to evaluate the treatments impact on preserving insulin production shortly after diagnosis. Participants will be followed for about 60 weeks to assess changes in key diabetes-related measures. Participants will be randomly assigned to receive either baricitinib or a placebo, both taken orally. The study compares these two groups to evaluate the effects of baricitinib on beta-cell function. The main measurement is the change in C-peptide area under the curve over 52 weeks, which indicates insulin production. Additional outcomes include changes in blood sugar control, insulin use, hypoglycemia events, and other health indicators. During the study, participants will attend visits for assessments and monitoring over approximately 60 weeks. Tests will include blood measurements like C-peptide and hemoglobin A1c, insulin dose tracking, and health surveys. Safety and drug levels will be monitored. Researchers will use these data to understand if baricitinib can help maintain beta-cell function in people newly diagnosed with type 1 diabetes.

This research aims to evaluate the long-term safety and effectiveness of the drug baricitinib in treating juvenile idiopathic arthritis JIA in children and adolescents aged 1 to under 18 years. Participants in this study have previously taken part in other baricitinib studies for JIA. The purpose is to monitor how well baricitinib works and to assess any serious side effects over an extended period. Participants will receive baricitinib orally during the study. This is a single-group trial where all participants receive the same treatment. The study follows participants for a total duration of up to 264 weeks approximately 5 years, during which they will be monitored closely for safety and disease activity. Throughout the study, participants will have regular assessments including monitoring for serious adverse events and treatment discontinuations. Researchers will also evaluate disease activity, pain levels, and other health measures using various clinical tools at baseline and at the end of the study period. This long-term follow-up ensures thorough evaluation of baricitinibs impact and participant well-being.

Juvenile psoriatic arthritis jPsA is a form of arthritis where the immune system attacks healthy joints, causing pain, stiffness, and swelling. This condition starts before age 16 and can worsen or improve over time. Researchers are studying how safe risankizumab is for treating jPsA and how it affects disease symptoms compared with adalimumab, which is already approved for this condition. This is a Phase 3 interventional trial enrolling about 40 children worldwide to evaluate these treatments. Participants are randomly assigned to receive either risankizumab or adalimumab given as subcutaneous injections based on their body weight. The study has two periods Period 1 lasts 24 weeks, where participants receive the assigned treatment. Those who respond continue with the same treatment for another 100 weeks in Period 2. Participants who worsen in Period 2 will stop the study treatment. After finishing treatment, there is a safety follow-up lasting 140 days for risankizumab and 70 days for adalimumab. During the study, participants will visit hospitals or clinics regularly for medical assessments, blood tests, and questionnaires to monitor treatment effects and side effects. Researchers will measure improvements using criteria such as the Juvenile Idiopathic Arthritis American College of Rheumatology response and track adverse events up to week 144. The total participation time includes treatment periods plus safety follow-up, lasting several years.

Researchers are evaluating the pharmacokinetics, pharmacodynamics, efficacy, and safety of intravenous anifrolumab compared with placebo in children aged 5 to under 18 years with moderate to severe active systemic lupus erythematosus SLE who are receiving standard care. This phase III clinical trial aims to better understand how anifrolumab works and its effects in this pediatric population. Participants are randomly assigned to receive either anifrolumab or matching placebo via intravenous infusion every 4 weeks. The study includes several periods Part A is a 4-week double-blind pharmacokinetic phase Part B is a 48- to 52-week double-blind safety and efficacy phase Part C is a 52-week open-label extension and Part D involves a safety follow-up visit 12 weeks after the last dose. During the approximately 116-week study, participants undergo screening for up to 30 days, followed by regular infusions and assessments including blood tests to measure drug levels and immune markers. Researchers evaluate responses using disease activity scores and track safety throughout. The study concludes with a safety follow-up visit to monitor participants after completing treatment.

Researchers are studying children aged 2 to under 18 years who have active juvenile enthesitis-related arthritis, including juvenile-onset ankylosing spondylitis, or juvenile psoriatic arthritis. The study aims to assess the levels of the drug bimekizumab in the blood after it is given by subcutaneous injection. This Phase 3 trial evaluates the safety and drug concentrations to better understand how bimekizumab behaves in pediatric participants with these specific types of juvenile arthritis. Participants will receive bimekizumab doses adjusted according to their weight, administered at specific times during the study. The initial treatment period lasts up to 16 weeks. The study also includes an extended safety follow-up of up to 141 weeks to monitor for any adverse events or side effects. No placebo or comparison group is used all participants receive the active drug. Throughout the study, participants will undergo regular assessments including blood tests to measure drug levels, vital signs, biochemistry and hematology parameters, growth measurements, and evaluations of arthritis disease activity. Researchers will also monitor adverse events, including specific infections and injection site reactions. The study tracks responses using standardized juvenile arthritis scores at Week 16. Participants are followed closely for safety and treatment effects throughout the treatment and follow-up periods, which may last several years.

Researchers are evaluating the effectiveness of apremilast compared with placebo for treating children aged 5 to less than 18 years with active juvenile psoriatic arthritis JPsA. This phase 3, double-blind, randomized, placebo-controlled study aims to assess how well apremilast works in improving symptoms and disease activity in pediatric participants with JPsA. Participants will be randomly assigned to receive either apremilast or a matching placebo orally during a 16-week double-blind treatment phase. After this period, all participants will continue with apremilast treatment for an additional 36 weeks in an open-label phase. The study will monitor the safety, efficacy, and pharmacokinetics of apremilast throughout these treatment periods. Participants will undergo regular assessments including evaluations of joint activity, pain levels, physician and participant disease activity ratings, and laboratory markers of inflammation. Researchers will measure responses using the American College of Rheumatology Pediatric ACR Pedi criteria and other disease activity scores. Safety monitoring includes tracking treatment-emergent adverse events and suicidal ideation. The study lasts a total of 52 weeks, with evaluations of growth and development included during the later weeks.

This research aims to improve treatment for children and adolescents newly diagnosed with lymphoblastic lymphoma. The trial is a large, international, randomized clinical study involving multiple countries and specialized centers. It focuses on whether using dexamethasone instead of prednisone during the induction phase can reduce relapses in the central nervous system CNS, and for high-risk patients, whether an intensified treatment improves event-free survival compared to standard therapy. Participants are grouped by risk level based on disease characteristics like CNS involvement and genetic markers. All patients first receive a prednisone prephase, then are randomized to either standard induction with prednisone or experimental induction with dexamethasone. Treatment phases include consolidation, extra-compartment phases adjusted by risk group, reintensification for some groups, and maintenance therapy lasting up to 24 months. Patients with CNS involvement receive intensified intrathecal therapy without cranial irradiation. High-risk patients may undergo a second randomization to receive either standard or intensified treatment during later phases. During the study, patients undergo various treatments including chemotherapy drugs such as cyclophosphamide, methotrexate, and PEG asparaginase. Researchers monitor disease relapse involving the CNS and measure event-free survival over a maximum of 7.25 years. Secondary assessments include overall survival, treatment-related toxicity and mortality, adverse events, and molecular markers. The trial involves ongoing follow-up for safety and effectiveness outcomes throughout the study period, which can last several years.